Annexon, Inc. · NASDAQ:ANNX
Annexon — Technical Presentation (February 2024)
17 slides from Annexon's technical presentation, Feb 10, 2024
Slide 1: Efficacy and Safety of Intravitreal ANX007 in Geographic Atrophy: ARCHER Phase 2 Results
Efficacy and Safety of Intravitreal Administration of ANX007 in Patients With Geographic Atrophy: Results of the ARCHER Phase 2 Study Eleonora Lad, Jeffrey Heier, Charles Wykoff, Glenn Jaffe, David Lally, Roger A. Goldberg, Allen Hu, Carl Regillo, David Boyer, Karl Csaky, Wendy Murahashi, Harman Han
Slide 2: Disclosures
Disclosures Scientific Advisor: 4-DMT, Alexion, Allegro, Annexon, Apellis, Aspen Neuroscience, Blue Rock, Boehringer Ingelheim, Broadwing Bio, Complement Therapeutics, Emmecell, Galimedix, IVERIC Bio/Astellas, Janssen, Kriya Therapeutics, LumiThera, Lutronic Vision, Nanoscope Therapeutics, NGM Bioph
Slide 3: Anti-C1q: A Unique Neuroprotective Mechanism
Anti-C1q: A Unique Neuroprotective Mechanism C1q initiates the classical complement cascade to drive photoreceptor synapse loss, cell loss and neuroinflammation • C1q is a key driver of neurodegeneration1 C1q binds stressed photoreceptor synapses and activates the classical pathway • C1q anchors cla
Slide 4: Photoreceptor Cells, Synapses & Function Are Lost Prior to RPE in GA
Photoreceptor Cells, Synapses & Function Are Lost Prior to RPE in GA Blocking C1q protects photoreceptor cells and function upstream of RPE loss Healthy Human Retina (top) • Uniform layer of photoreceptor synapses (red) and photoreceptor neurons (blue) GA Patient Retina (Bottom) Healthy Healthy huma
Slide 5: Anti-C1q Protected Photoreceptor Cells and Their Function in Models of Photoreceptor Damage
Anti-C1q Protected Photoreceptor Cells and Their Function in Models of Photoreceptor Damage C1q Deposition on Photoreceptor Cells Anti-C1q Protected Photoreceptors and Function and Synapses with Light-Induced Damage ANTI-C1Q PROTECTED 3 DAYS POST WHITE PHOTORECEPTOR CELLS/ PROTECTED RETINAL CONTROL
Slide 6: ARCHER: ANX007 Phase 2 Trial in GA
ARCHER: ANX007 Phase 2 Trial in GA Randomized, double-masked Included foveal and non-foveal lesions Stratified for lesion location and lesion size 12 months (n=270) Sham ANX007 ANX007 monthly or every other month 5mg monthly (EM) 5mg every other month (EOM) (n=89) (n=89) (n=92) PRIMARY BIOMARKER END
Slide 7: ANX007 Demonstrated Statistically Significant Protection From Vision Loss as Measured by BCVA
ANX007 Demonstrated Statistically Significant Protection From Vision Loss as Measured by BCVA ≥15-Letter Loss PATIENTS WITH PERSISTENT BCVA ≥15-LETTER LOSS THROUGH MONTH 12# 25 21.3% 5.6% 10.9% 20 • First known significant preservation of vision in GA % Patients 15 10 • Dose-dependent response infor
Slide 8: Significant, Time-Dependent Protection From ≥15 Letter Vision Loss with Monthly Treatment
Significant, Time-Dependent Protection From ≥15 Letter Vision Loss with ANX007 Monthly Treatment BCVA ≥15-LETTER LOSS AT 2 CONSECUTIVE VISITS THROUGH MONTH 12 OR LAST VISIT 100% % Patients without ≥15 Letter BCVA Loss 95% EM (n=89) 72% Risk Reduction ANX007 EM 90% HR (CI) = 0.28 (0.11 to 0.76) p = 0
Slide 9: BCVA Subgroup Analysis: Foveal and Non-Foveal Patients
ANX007 BCVA Subgroup Analysis: Protection from Vision Loss in Foveal and Non-Foveal Patients PATIENTS WITH PERSISTENT ≥15-LETTER LOSS THROUGH MONTH 12# 30 25.0% 5.9% 18.4% 17.8% 5.3% 2.3% Sham ANX007 EM 20 ANX007 EOM % Patients Nominal summary p- 10 value vs. sham^ ANX007 EM 0.0019 11/44 3/51 9/49 8
Slide 10: BCVA ≥15-Letter Loss Accelerates After Cessation of Treatment
BCVA ≥15-Letter Loss Accelerates After Cessation of Treatment Visual Function Loss Parallels Sham in Off-Treatment Period % PATIENTS WITH ANY BCVA ≥15-LETTER LOSS FROM BASELINE 30 Off Treatment • Low frequency (<10% per Sham Pooled (n=89) timepoint) of single BCVA 25 ANX007 EOM (n=92) ANX007 EM (n=8
Slide 11: ANX007 Did Not Significantly Reduce GA Lesion Area
ANX007 Did Not Significantly Reduce GA Lesion Area GA LESION AREA CHANGE FROM BASELINE AT MONTH 12# 2.15 2.02 2.12 LS Mean（±SE）Change 2.5 6% decrease 2.0 from Baseline, mm2 1.5 1.0 0.5 n = 89 n = 89 n2.12 = 92 0 Sham ANX007 ANX007 EM EOM Nominal 3 6 9 12 p-value vs --- 0.526 0.896 sham^ Months #The
Slide 12: Ellipsoid Zone (EZ) Change Directly Measures Photoreceptor Anatomy Rather than RPE
Ellipsoid Zone (EZ) Change Directly Measures Photoreceptor Anatomy Rather than RPE Protection of photoreceptors may arise from synaptic protection ARCHER EZ Population OCT • 194 patients with OCT scans from Heidelberg Spectralis Sham ANX007 EM ANX007 EOM Total 72 61 61 194 Ellipsoid Zone (EZ): from
Slide 13: Similar Protection from Vision Loss Across Total ARCHER Subjects and EZ Population
Similar Protection from Vision Loss Across Total ARCHER Subjects and EZ Population Total ARCHER Cohort EZ Population Only PATIENTS WITH PERSISTENT BCVA PATIENTS WITH PERSISTENT BCVA ≥15-LETTER LOSS THROUGH MONTH 12# ≥15-LETTER LOSS THROUGH MONTH 12# 25 25 23.6% 3.3% 9.8% 21.3% 5.6% 10.9% 20 20 % Pat
Slide 14: Initial Assessment of Ellipsoid Zone Change: ~30% Change from Sham in EZ Attenuation
Initial Assessment of Ellipsoid Zone Change: ANX007 Demonstrated ~30% Change from Sham in EZ Attenuation As observed 12-month completer data; MMRM analysis ongoing, pending full timepoint assessment TOTAL EZ ATTENUATION (EZ = 0 mm) PARTIAL EZ ATTENUATION (EZ < 20 mm) CHANGE FROM BASELINE AT MONTH 12
Slide 15: ANX007 Generally Well-Tolerated
ANX007 Generally Well-Tolerated ADVERSE EVENTS OF SPECIAL INTEREST SHAM ANX007 EM ANX007 EOM n (%) (N=89) (N=89) (N=92) 3 4 4 Choroidal Neovascularization (3.4%) (4.5%) (4.3%) 1 2 Endophthalmitis 0 (1.1%) (2.2%) INTRAOCULAR INFLAMMATION DETAILS* n 1^ Iritis – 1 Retinal Vascular Occlusion 0 0 (1.1%)
Slide 16: ANX007 Global GA Pivotal Program to begin Mid-2024
ANX007 Global GA Pivotal Program to begin Mid-2024 Replicate ARCHER and support global approvals PRIME Designation ARCHER II TRIAL Primary analysis: Treatment masked from EMA 12 months through 24 months** Sham Study* ANX007 Monthly (n = ~200) Sham (n = ~200) Primary Endpoint Persistent ≥15-Letter BC
Slide 17: ANX007: A Novel Neuroprotective Agent Demonstrating Benefit in Vision in ARCHER Trial
ANX007: A Novel Neuroprotective Agent Demonstrating Benefit in Vision in ARCHER Trial • ANX007’s unique mechanism of action targets C1q pathway to protect photoreceptors • ANX007 demonstrated consistent visual function benefits Highly statistically significant on visual acuity endpoint Dose and